Abstract
Recombinant adeno-associated virus (rAAV) vector is one of the promising delivery tools for gene therapy. Currently, hundreds of clinical trials are performed but the major barrier for clinical application is the absence of any ideal large scale production technique to obtain sufficient and highly pure rAAV vector. The large scale production technique includes upstream and downstream processing. The upstream processing is a vector package step and the downstream processing is a vector purification step. For large scale downstream processing, the scientists need to recover rAAV from dozens of liters of cell lysate or medium, and a variety of purification strategies have been developed but not comprehensively compared till now. Consequently, this review will evaluate the scalable downstream purification strategies systematically, especially those based on the physicochemical properties of AAV virus, and attempt to find better scalable downstream strategies for rAAV vectors.
Keywords: Clinical application, downstream purification, large scale, physicochemical properties, purification strategy, rAAV vector.
Current Pharmaceutical Biotechnology
Title:Scalable Downstream Strategies for Purification of Recombinant Adeno- Associated Virus Vectors in Light of the Properties
Volume: 16 Issue: 8
Author(s): Weihong Qu, Mingxi Wang, Yaqing Wu and Ruian Xu
Affiliation:
Keywords: Clinical application, downstream purification, large scale, physicochemical properties, purification strategy, rAAV vector.
Abstract: Recombinant adeno-associated virus (rAAV) vector is one of the promising delivery tools for gene therapy. Currently, hundreds of clinical trials are performed but the major barrier for clinical application is the absence of any ideal large scale production technique to obtain sufficient and highly pure rAAV vector. The large scale production technique includes upstream and downstream processing. The upstream processing is a vector package step and the downstream processing is a vector purification step. For large scale downstream processing, the scientists need to recover rAAV from dozens of liters of cell lysate or medium, and a variety of purification strategies have been developed but not comprehensively compared till now. Consequently, this review will evaluate the scalable downstream purification strategies systematically, especially those based on the physicochemical properties of AAV virus, and attempt to find better scalable downstream strategies for rAAV vectors.
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Cite this article as:
Qu Weihong, Wang Mingxi, Wu Yaqing and Xu Ruian, Scalable Downstream Strategies for Purification of Recombinant Adeno- Associated Virus Vectors in Light of the Properties, Current Pharmaceutical Biotechnology 2015; 16 (8) . https://dx.doi.org/10.2174/1389201016666150505122228
DOI https://dx.doi.org/10.2174/1389201016666150505122228 |
Print ISSN 1389-2010 |
Publisher Name Bentham Science Publisher |
Online ISSN 1873-4316 |
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